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A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

PHASE2RECRUITING

The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.

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Study details:

This is a Phase 2a, multi-center, randomized, open-label study of sapablursen in up to 40 participants with PD-PV. The study consists of 4 periods: 1) Screening Period: up to 7 weeks; 2) Treatment Period: 37 weeks 3) Treatment Extension Period: 36 weeks; 4) Post-treatment Period: 13 weeks. In the Treatment Period, study drug is given by subcutaneous (under the skin) injection(s).

There will be a total of 9 doses given over about 8 months. In the Treatment Extension Period, there will be a total of 9 doses given over about 8 months. Participants will be assigned to receive one of 2 Dosing Levels - a higher or a lower level, with an equal chance of being assigned to either Dosing Level.

All participants will receive study drug; there is no placebo. This study was extended to allow participants to receive sapablursen for an additional 36 weeks following the initial 37-week treatment period.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis

Participant must be phlebotomy dependent.

Participants do not need to be on cytoreductive therapy and do not need to have been previously treated with cytoreductive therapy. If the patient was previously on cytoreductive therapy it must have been discontinued at least 3 months prior to Screening, with all associated AEs resolved. If the patient is currently on cytoreductive therapy they must be on a stable dose of hydroxyurea, recombinant or PEGylated interferon, or ruxolitinib for at least 3 months prior to Screening.

Exclusion criteria

Meets criteria for post-polycythemia vera myelofibrosis (PPV-MF) as defined by the International Working Group- Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)

Moderate to severe splenic pain or spleen-related organ obstruction

Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP)

Known primary or secondary immunodeficiency

Active infection with human immunodeficiency virus (HIV), hepatitis C, or hepatitis B.

Active infection requiring systemic antiviral or antimicrobial therapy or active novel coronavirus disease (Covid-19) infection

Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or non-metastatic prostate cancer that has been successfully treated

Surgery requiring general anesthesia within 1 month prior to Screening

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2021-12-30

Primary completion: 2025-01-01

Study completion finish: 2025-12-01

Study type

TREATMENT

Phase

PHASE2

Trial ID

NCT05143957

Intervention or treatment

DRUG: sapablursen

Conditions

• Phlebotomy Dependent Polycythemia Vera

Image related to Phlebotomy Dependent Polycythemia Vera

Condition: Phlebotomy Dependent Polycythemia Vera
DRUG: sapablursen
Blacktown, New South Wales, Australia and more
Sponsor: Ionis Pharmaceuticals, Inc.

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Blacktown Hospital

Research sites nearby

Select from list below to view details:

Blacktown Hospital
Blacktown, New South Wales, Australia
St. George Hospital
Kogarah, New South Wales, Australia
Calvary Mater Newcastle Hospital
Waratah, New South Wales, Australia
Border Medical Oncology Research Unit
Albury, Not Specified, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Sapablursen Dose Level 1 Sapablursen will be administered by SC injection every 4 weeks.	DRUG: sapablursen Sapablursen will be administered by SC injection.
EXPERIMENTAL: Sapablursen Dose Level 2 Sapablursen will be administered by SC injection every 4 weeks	DRUG: sapablursen Sapablursen will be administered by SC injection.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period	Not Specified	Week 17 to Week 37

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Proportion of patients achieving a reduction in the frequency of phlebotomy by ≥ 30%, ≥ 50%, ≥ 75% and ≥ 90% comparing Baseline with the last 20 weeks of the 37-week Treatment Period	Not Specified	Week 17 to Week 37
Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37	Not Specified	Baseline up to Week 37

Frequently Asked Questions

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

Clinical Trials Gov: A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera